H3B-8800, an orally available small-molecule inhibitor targeting SF3B1, selectively eradicates SF3B1-mutant leukemia cells in xenografts (Seiler et al., 2018) and is currently been investigated in a phase 1/2 clinical trial in AML and MDS (Table 1). The gene discussed is SF3B1; the disease is acute myeloid leukemia.